A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia (eTHINK)

November 21, 2019 updated by: Novo Nordisk A/S

Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)

The Hemophilia Growth and Development Study (HGDS) nearly 25 years ago showed haemophilia and HIV impacted brain development, thinking, and behaviour in children and adolescents. The eTHINK study is designed to understand whether advances in hemophilia treatment have removed any impact of hemophilia. If there still is an impact of hemophilia, the eTHINK study will help to identify which children or adolescents are still at risk, and how to screen other children. Participants and their child will participate in a comprehensive assessment of child's brain development, thinking and behavior through completing tests and questionnaires. There is only one study visit of 1 to 1.5 hours with no follow-up required

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

563

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Novo Nordisk Investigational Site
      • Orange, California, United States, 92868
        • Novo Nordisk Investigational Site
      • San Diego, California, United States, 92123
        • Novo Nordisk Investigational Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Novo Nordisk Investigational Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20010-2978
        • Novo Nordisk Investigational Site
    • Florida
      • Tampa, Florida, United States, 33607
        • Novo Nordisk Investigational Site
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Novo Nordisk Investigational Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Novo Nordisk Investigational Site
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Novo Nordisk Investigational Site
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Novo Nordisk Investigational Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Novo Nordisk Investigational Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • Novo Nordisk Investigational Site
      • Detroit, Michigan, United States, 48201
        • Novo Nordisk Investigational Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Novo Nordisk Investigational Site
    • Nebraska
      • Omaha, Nebraska, United States, 68198-6828
        • Novo Nordisk Investigational Site
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Novo Nordisk Investigational Site
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Novo Nordisk Investigational Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Novo Nordisk Investigational Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19134
        • Novo Nordisk Investigational Site
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Novo Nordisk Investigational Site
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Novo Nordisk Investigational Site
    • Washington
      • Seattle, Washington, United States, 98105
        • Novo Nordisk Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 21 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

Children and young adults with hemophilia A or B, all severities

Description

Inclusion Criteria: - Male child or young adult (ages 1:0-3:6 and 4:0-21:11 years with diagnosis of congenital hemophilia A or B, any severity, with or without inhibitors - Parent able and willing to provide consent, and young adult with hemophilia (age 18-21) able to provide consent; children (ages 7-14) and adolescents/young adults (ages 14-17) able and willing to provide assent based upon local institutional policies - Parent able and willing to complete neurodevelopment and cognitive parent reported scales during comprehensive or other Hemophilia Treatment Center visit for about 45-60 minutes in English or Spanish depending on age group) - Children, adolescents and young adults with hemophilia (ages 1-21) able to participate in assessments of development and intelligence for about 40-45 minutes and adolescents and young adults (age 11-21) with hemophilia able and willing to complete additional self-assessment scales for about 30-45 minutes during comprehensive or other elective visit for in English or Spanish (depending on age group) - Provision of informed consent before the start of any study-related activities Exclusion Criteria: - Patient aged 3:7-3:11 years (43-47 months) - Prior participation in the study - Prior neurocognitive screening with the same or related instruments in the prior 6 months - Patients with hemophilia or their caregivers for whom test measures may be culturally inappropriate - Patients with hemophilia with known history of alcohol or substance abuse - Patients with hemophilia who have used opiates in the past 24 hours or other illicit drugs within the past 48 hours - Patients with hemophilia currently experiencing an acute bleed that has not resolved - Patients with hemophilia with recent head injury or concussion within the past 4 weeks - Concurrent diagnosis of another bleeding or thrombotic disorder - Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with hemophilia
Children and young adults with hemophilia A or B, all severities, treated in the hemophilia treatment center (comprehensive care) setting, including participation of caregivers/parents completing questionnaires designed to evaluate neurologic, neurocognitive and neurobehavioral function and development.
Other: No treatment given
The participants will undergo a brief physical examination focused on neurologic function (muscle function, sensation, coordination, walking). Parents will be asked by the study team or the psychologist about their children and will complete several commonly used standard surveys about brain development, thinking, behavior, and decision making. The participants will also engage with the psychologist to evaluate development or thinking (IQ), and depending on their age they may be asked to complete one or more commonly used standard surveys about behavior and decision making.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cognition composite score according to the BAYLEY-III instrument
Time Frame: Day 1
Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average
Day 1
Behavioural Symptoms Index according to Behavior Assessment System for Children - Third Edition (BASC-3) parent rating scale
Time Frame: Day 1
Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average
Day 1
Adaptive behaviour domain score according to Adaptive Behavior Assessment System, Third Edition (ABAS-3) rating scale
Time Frame: Day 1
Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average
Day 1
Global executive composite index according to Behavior Rating Inventory of Executive Function (BRIEF)
Time Frame: Day 1
Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average
Day 1
Attention and processing speed (full scale IQ) according to Wechsler Preschool and Primary Scale of Intelligence-4th Edition (WPPSI-IV) for age group 4-6 years or Wechsler Abbreviated Scale of Intelligence, 2nd Edition (WASI-II) for age group 7-22 years
Time Frame: Day 1
Normalized score compared to US population (0 = equivalent to US average, negative = below US average, positive = above US average
Day 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novo Nordisk A/S

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

September 27, 2018

Primary Completion (ACTUAL)

October 11, 2019

Study Completion (ACTUAL)

October 11, 2019

Study Registration Dates

First Submitted

August 21, 2018

First Submitted That Met QC Criteria

September 6, 2018

First Posted (ACTUAL)

September 7, 2018

Study Record Updates

Last Update Posted (ACTUAL)

November 22, 2019

Last Update Submitted That Met QC Criteria

November 21, 2019

Last Verified

November 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HAEM-4436
  • U1111-1202-3415 (OTHER: World Health Organization (WHO))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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